World Orphan Drug Congress 2026: Advancing Rare Disease Therapies Through Collaboration

The World Orphan Drug Congress convenes pharmaceutical and biotechnology companies, regulators, patient advocates, payers, investors, and solution providers to advance orphan drug development and improve access to therapies for rare disease patients. Scheduled for June 9–11, 2026, at the Thomas M. Menino Convention & Exhibition Center in Boston, Massachusetts, the event combines a workshop day with two days of conference sessions and an exhibition. Its central themes center on breaking barriers and fostering collaboration, with organizers aiming to accelerate innovation, strengthen partnerships, and address persistent challenges in diagnosis, development, and patient access.

Lifecycle-Focused Content Streams

Sixteen distinct content streams structure the program around the full lifecycle of orphan drug development. Sessions address patient data and real-world evidence practices, next-generation therapies, payer strategies, clinical development and regulatory pathways, commercial launch approaches, rare disease advocacy, global market access, pricing and reimbursement models, investor matchmaking, advanced therapy manufacturing and clinical development, rare oncology, commercialization of cell and gene therapies, digital health and artificial intelligence applications, diagnostic improvements, and early-stage discovery technologies. These streams integrate keynote-driven presentations with panel discussions and facilitated roundtables, providing attendees with targeted opportunities to examine data ownership, decentralized trials, innovative contracting, and newborn screening expansion. The exhibition complements the agenda by showcasing products and services from more than 100 participating organizations.

Diverse Speaker Lineup

More than 280 speakers drawn from industry, government, academia, and patient communities anchor the program. Participants include executives from Johnson & Johnson, Sanofi, Sarepta Therapeutics, Amgen, and Takeda; officials from the NIH/NCATS and FDA; leaders of patient organizations such as Global Genes, the National Organization for Rare Disorders, and the Immune Deficiency Foundation; and representatives from investment firms and contract research organizations. Their affiliations span regulatory science, market access, clinical development, patient advocacy, and venture capital, creating balanced representation across stakeholder groups. This diversity supports cross-sector dialogue on topics ranging from reimbursement policy to gene therapy commercialization.

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Shaping Future Access Policies

Discussions across the sixteen streams are positioned to shape collaborative standards for data sharing, regulatory flexibility, and access frameworks that directly affect rare disease communities. By convening payers alongside developers and advocates, the World Orphan Drug Congress may inform evolving approaches to pricing one-time therapies and negotiating outcomes-based contracts. Emphasis on advanced therapy manufacturing and digital tools could accelerate scalable production models and patient-finding capabilities, while advocacy-focused sessions may strengthen policy initiatives around newborn screening and decentralized trials. The event’s matchmaking and networking components further support partnership formation that sustains innovation pipelines and improves therapy availability for underserved patient populations.